This is a placebo-controlled clinical trial to assess the utility of Guanfacine Extended Release (GXR) in the management of patients with Prader Willi Syndrome (PWS) who have significant aggression or self-injury. The purpose of this trial is to establish the safety of GXR with a specific focus on metabolic effects.
Prader-Willi syndrome is a genetic disorder due to loss of function of specific genes. In newborns, symptoms include weak muscles, poor feeding, and slow development. Beginning in childhood, the person becomes constantly hungry, which often leads to obesity and type 2 diabetes. Also, mild to moderate learning disability and behavioral problems are typical. Guanfacine Extended Release (GXR), the investigational drug in this study would be the first study to evaluate the drug in patients with Prader Willi Syndrome. "Investigational" means it is not approved by the Food and Drug Administration (FDA) to treat Prader Willi Syndrome. However, Guanfacine Extended Released (GXR) is an FDA approved drug used to treat children and adolescents with hypertension and attention deficit hyperactivity disorder (ADHD). GXR is thought to respond to parts of the brain that lead to strengthening working memory, reducing distraction, improving attention and impulse control. GXR is generally considered safe for children as long as it is used according to the dosing instructions (up to 4mg) of a qualified medical professional.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
DOUBLE
Placebo will be administered same times as GXR
The starting dose for all subjects will be 1 mg per day. If the medication is well-tolerated, the dose can be raised to 2 mg until day 28 and increased to 3mg for the remaining 4 weeks in the trial. The dose schedule will not be fixed; the treating clinician can delay a planned increase or lower the dose to manage adverse effects. At week 8, the study will be unblinded and subjects will continue treatment for 8 more weeks.
NYU Langone Health
New York, New York, United States
CGI-I scale rating
A positive clinical response will be determined by a rating of 1 or 2 (Very much/Much improved) on the CGI-I scale at the end of the blinded trial.
Time frame: 19 Weeks
Aberrant Behavior Checklist
Consists of 2 subscales; irritability (15 items) and hyperactivity/noncompliance (16 items).
Time frame: 19 Weeks
Self-Injury Trauma scale
SHI scores of 5 or greater were found to be indicative of borderline personality disorder. Part 1 is ranking based on the number of wounds 1=one would (common in a mild SIB but rare in a severe case) 2=two or four wounds (common) and 3=five or more wounds (rare). Injury severity is scored on a subjective basis with labels such as "mild" "moderate" and "severe" accompanied by descriptions of the observed state of the anatomy. Part 3 is the Estimate of Current Risk.
Time frame: 19 Weeks
Modified Overt Aggression Scale
four-part behavior rating scale used to evaluate and document the "frequency and severity" of aggressive episodes.\[1\] The rating scale is made up of four categories; verbal aggression, aggression against objects, aggression against self, and aggression against others
Time frame: 19 Weeks
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